Pompe Empowerment Program (PEP) 2022
Starting Jan 16, 2022, this online series brings together Pompe disease families and other experts in five 2-hr sessions over 11 days.
Avalglucosidase Alfa (Nexviazyme™) approved by Health Canada
Health Canada has approved avalglucosidase alfa (Nexviazyme™) for the long-term treatment of late-onset Pompe disease in patients over the age of 6 months.
FDA approves Nexviazyme® (avalglucosidase alfa-ngpt)
In a recent announcement, the Pompe disease community learned that the U.S. Food and Drug Administration (FDA) has approved Nexviazyme® for the treatment of late-onset Pompe patients that are one year old and older.
Amicus Remains Hopeful about AT-GAA Despite Mixed Results from Phase 3 Study
Amicus Therapeutics recently announced topline results of its phase 3 PROPEL study of its combination therapy, AT-GAA, for the treatment of late onset Pompe disease (LOPD).
New Review Article Highlights Latest Developments in Pompe Disease Research
An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
New ERT For Late-onset Pompe Disease Shows Promise
Results from the COMET study were recently reported showing neoGAA (avalglucosidase alfa), the newer enzyme replacement therapy (ERT) currently in development, to be as effective as the currently approved ERT, Myozyme (alglucosidase alfa), in persons with late-onset Pompe disease.
Sanofi Genzyme COVID-19 Update
At this time, Sanofi Genzyme does not anticipate shortages for patients resulting from the COVID-19 situation.
Amicus Therapeutics’ Clinical Trials During the COVID-19 Outbreak
Patients enrolled in all clinical studies for AT-GAA for Pompe disease, including the PROPEL pivotal study, will continue to receive study drug.
Amicus Therapeutics Provides Updates for AT-GAA for Pompe Disease
Amicus Therapeutics today announced clinical, regulatory and manufacturing advancements for AT-GAA.
Amicus Therapeutics Receives Breakthrough Therapy Designation for Pompe Treatment
Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease.